National Academy of Sciences, Proceedings of the National Academy of Sciences, 52(116), p. 26846-26852, 2019
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Significance This study demonstrates a therapeutic option using induced pluripotent stem cells (iPSCs), gene editing, and tissue engineering techniques for the development of a long-lasting treatment that will result in the permanent closure of nonhealing wounds in dystrophic epidermolysis bullosa (DEB), especially for recessive DEB (RDEB). Here, we demonstrated that the combination of iPSCs, gene editing, and tissue engineering allows us to perform efficient gene correction by homology-directed repair with the CRISPR-Cas9 gRNA ribonucleoprotein complex system, differentiate the gene-corrected RDEB patient iPSCs into skin cells, and generate human skin equivalents (HSEs) that show skin integrity and type VII collagen restoration after grafting them onto nude mice. The results from this study serve as a foundation to translate this treatment into the clinic.